Pharmacological Chaperones for the Mucopolysaccharidoses

Abstract excerpt

The mucopolysaccharidoses (MPS) are a group of 11 lysosomal storage diseases (LSDs) produced by mutations in the enzymes involved in the lysosomal catabolism of glycosaminoglycans. Most of the mutations affecting these enzymes may lead to changes in processing, folding, glycosylation, pH stability, protein aggregation, and defective transport to the lysosomes. The use of small molecules, called pharmacological cha...

Topics

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• Gaucher Disease
• Lysosomal Storage Diseases
• Lysosomal Storage Disorders Research
• Molecular Chaperones
• Mucopolysaccharidosis II
• Mucopolysaccharidosis IV
• Mutation
• Protein Folding

Identifiers and source

Literature Corpus work

ffc0732c-4917-560f-bb6a-cf4f1a83649a

DOI

10.20944/preprints201910.0365.v1